PROJECT(ABSTRACT ? 2019 Congenital Muscle Disease Scientific Symposium Cure CMD (Cure Congenital Muscular Dystrophy) seeks support from the National Institutes of Health to collaborate with aligned patient advocacy organizations A Foundation Building Strength (AFBS, focused on Nemaline Myopathy or NM) and Team Titin (focused on Titinopathy or TTN), in order to hold a two-part Scientific and Family Conference, to be held on July 25-28, 2019. Having recognized significant overlap in symptomatology, care management, and research communities, representatives from each organization believes they will benefit significantly from dialogue, information exchange, and collaboration. The ultimate aim is to accelerate progress toward clinical trials, treatments, and eventually, a cure for congenital muscle disease. Part one of the conference will be a Scientific Symposium (for which Cure CMD seeks support from the National Institutes of Health) that will include patient community representatives from each subtype; part two of the conference will be a Family Conference, wherein scientists will educate, learn from, and mingle with affected individuals and families. Key objectives are to: - Create, shape, and refine research projects and plans with clear patient input on need and design. At the conference, scientists working in congenital muscle disease will review proposed and current research projects and welcome challenges, support, and feedback from the scientific and patient communities; - Launch innovative, responsive research projects that reflect community needs, build on new collaborations, and engage the patient community; - Increase available care information to patients, caregivers, and clinicians, strategizing ways to avail valuable, life-changing care guidelines and strategies; and - Draft Subtype-Specific Plans for Research for the next three years, offering a roadmap to benefit the community; and - Ultimately, speed implementation of treatments and improve health outcomes. We believe collaboration is a critical step in speeding up practical knowledge and discoveries, and in utilizing evidence across the healthcare continuum. With a recently completed clinical trial in congenital muscular dystrophy declared a success, a natural history study for NM planned for launch in 2019, significant foundational research in TTN completed this year, and promising, relevant new research projects underway in all three conditions, this triad of collaborators?Cure CMD, A Foundation Building Strength (AFBS), and Team Titin? is eager to come together at this pivotal time in the story of congenital muscle disease.